Our latest research, all in one place. Browse our collection of journal articles, reports and conference proceedings to see how we’re contributing to HEOR research. Remember to:
BACKGROUND: Highly active (HA) relapsing remitting multiple sclerosis (RRMS) is associated with frequent relapses and high burden of disease/disability. Natalizumab is licensed for HA RRMS, including rapidly evolving severe (RES) (=2 relapses in previous year) and sub-optimally treated (SOT) (=1 relapse in previous year despite treatment) populations. However, there is limited RCT evidence in the SOT subpopulation.
OBJECTIVE: To review the non-RCT evidence for natalizumab in SOT HA RRMS.
METHODS: Databases were searched to January 2023 for non-randomised studies of natalizumab in HA RRMS. Studies in patients with =1 relapse during previous treatment were eligible for inclusion. Meta-analyses were conducted to compare natalizumab with platform and higher efficacy disease-modifying therapies, with sensitivity analysis restricted to studies of low risk of bias.
RESULTS: Included comparative studies (n = 16) showed natalizumab had lower relapse rates, disease activity and MRI (radiological) outcomes compared with platform and higher efficacy therapy. Case series (n = 11) showed natalizumab was associated with high rates of freedom from relapse and clinical/radiological disease activity and reductions in annualised relapse rate and disability progression.
CONCLUSIONS: Literature reviewed indicates that natalizumab is more effective than other included treatments for SOT patients. Findings were consistent with studies in the broad HA RRMS population, suggesting that natalizumab may have similar efficacy for SOT and RES HA RRMS.
INTRODUCTION: Gastroesophageal reflux disease (GERD) is a common ailment associated with troublesome symptoms. The standard of care in Italy involves initial treatment with proton pump inhibitor (PPI)-based medical management or laparoscopic Nissen fundoplication (LNF) for patients unwilling to continue or intolerant of long-term PPI therapy. RefluxStop is a novel medical device, intended for laparoscopic implantation, that has recently proven to be an efficacious and cost-effective treatment option for patients with GERD. This analysis aims to describe the short-term budget impact of introducing RefluxStop as a GERD treatment option within the Italian National Health Service (SSN).
METHODS: A model adherent to international best practice recommendations was developed to estimate the budget impact of introducing RefluxStop over a 5-year time horizon. Two scenarios were considered: one without RefluxStop (i.e., comprising PPI therapy, LNF, and magnetic sphincter augmentation using the LINX system); and one with RefluxStop (i.e., addition of RefluxStop to the three treatment options previously mentioned). Clinical benefits and costs associated with each intervention were included in the analysis.
RESULTS: Over 5 years, the introduction of RefluxStop resulted in avoidance of 95 surgical failures, 11 reoperations, and 64 endoscopic esophageal dilations. Introduction of RefluxStop resulted in an almost neutral impact on the existing budget with a 0.316% increase in the annual Italian SSN spending on GERD treatment.
CONCLUSION: Introduction of RefluxStop as a GERD treatment option in Italy is likely to be associated with substantial clinical benefits and a marginal budget impact.
INTRODUCTION: Three randomised controlled trials (RCTs) have demonstrated that first-line cryoballoon pulmonary vein isolation decreases atrial tachycardia in patients with symptomatic paroxysmal atrial fibrillation (PAF) compared with antiarrhythmic drugs (AADs). The aim of this study was to develop a cost-effectiveness model (CEM) for first-line cryoablation compared with first-line AADs for the treatment of PAF. The model used a Danish healthcare perspective.
METHODS: Individual patient-level data from the Cryo-FIRST, STOP AF and EARLY-AF RCTs were used to parameterise the CEM. The model structure consisted of a hybrid decision tree (one-year time horizon) and a Markov model (40-year time horizon, with a three-month cycle length). Health-related quality of life was expressed in quality-adjusted life years (QALYs). Costs and benefits were discounted at 3% per year. Model outcomes were produced using probabilistic sensitivity analysis.
RESULTS: First-line cryoablation is dominant, meaning it results in lower costs (-€2,663) and more QALYs (0.18) when compared to first-line AADs. First-line cryoablation also has a 99.96% probability of being cost-effective, at a cost-effectiveness threshold of €23,200 per QALY gained. Regardless of initial treatment, patients were expected to receive ~ 1.2 ablation procedures over a lifetime horizon.
CONCLUSION: First-line cryoablation is both more effective and less costly (i.e. dominant), when compared with AADs for patients with symptomatic PAF in a Danish healthcare system.
BACKGROUND: Single use health technologies are known to be approximately 50% more environmentally harmful than their reusable equivalent [1-3]. This novel pilot study was undertaken for the Scottish Health Technologies Group (SHTG) and is the first UK published parallel assessment alongside a cost effectiveness evaluation [4]. The aim of this research was to use hybrid and innovative methodological approach to evaluate environmental impact, utilising established multidisciplinary methods, in conjunction with HTA principles.
METHODS: Through the application of environmental management principles and reporting, the Greenhouse Gas (GHG) Protocols [5], ISO14040 [6] and Publicly Available Specification (PAS) 2050 [7] were applied in combination with overarching HTA principles. An environmental life cycle assessment (LCA) of single use rhinolaryngoscopes was compared with a reusable equivalent. A hybrid approach was used to collect data to quantify carbon intensity including, process activity data, real world data and secondary data. Data were extrapolated to estimate national GHG emissions and waste volumes to support NHS sustainability targets. The carbon output per functional unit were categorised using thresholds based on PAS recommendations to strengthen support for the HTA decision making process. Sensitivity analyses were performed to overcome data issues. Further recommendations were made support NHS net zero targets, promoting the use of more sustainable health technologies along the healthcare pathway.
RESULTS: The carbon emissions of a single use flexible rhinolaryngoscope compared to its reusable equivalent was 6.03kgCO2e and 3.26kgCO2e respectively. The findings of the report are in line with research reporting similar carbon emissions and provide important methods for HTA. Data extrapolation (2264 procedures per annum) reveals the annual environmental impact of this technology, throughout NHS Scotland, to be 13,652kgCO2e and 7,381kgCO2e, for the single use and reusable devices, respectively. Raw material acquisition, the use of personal protective equipment and transportation were highlighted as carbon hotspots along the healthcare pathway. The estimated waste volumes are 12.58 tonnes and negligible volumes of clinical waste per annum for the single use versus reusable equivalent, respectively.
LIMITATIONS: A comparable LCA relies on a solid foundation of data. However, a high-level life cycle inventory based on good quality data was not possible for this parallel assessment, mainly attributed to the lack of access to primary data sources; influenced by a lack of regulation. Consequently, there was heavy reliance on secondary data and therefore the findings of this study may lack robustness and should be considered with caution.
IMPLICATIONS: The findings of this study support health technology environmental sustainability policy and framework development. This study recommends a hybrid methodological approach to evaluate environmental impact of health technologies. This approach supports product LCA [8] and carbon hotspot evaluation. This research also supports valuable data generation in a field nascent of research to support methodological development [9-11]. According to literature, this is the first parallel assessment as part of a HTA to be published [10]. Environmental outcomes used in the pilot study, align with government and NHS England net zero and waste reduction objectives [12]. This pilot study focuses on reporting GHG emissions. This is reasonable given that climate change and planetary health is determined largely by anthropogenic GHG emissions; fundamental for the stability of other environmental systems [13], and that national targets are focused on GHG emissions [12]. However, measuring GHG emissions and waste volumes only, can be considered a disadvantage, in that other important environmental impacts across other areas may be overlooked [1, 2, 14]. Therefore, future research should undertake a broader environmental assessment.
This systematic review and meta-analysis was conducted to assess the randomized controlled trial (RCT) evidence available for renal denervation (RDN) in uncontrolled arterial hypertension. Twenty-five RCTs met the eligibility criteria for the systematic review, and 16 RCTs were included in the meta-analysis. The results of the random effects meta-analysis estimated a mean difference of -8.5 mmHg [95% confidence interval (CI) -13.5 to -3.6] for office SBP, -3.6 mmHg (95% CI -5.2 to -2.0) for 24 h SBP and -3.9 mmHg (95% CI -5.6 to -2.2) for ambulatory daytime SBP in favour of RDN compared with control (medication and/or sham-only) at primary follow-up. Similarly favourable results were observed across a range of prespecified subgroup analyses, including treatment-resistant hypertension. This meta-analysis suggests that the use of RDN in uncontrolled hypertension leads to consistent reductions in blood pressure. Reductions appear to be statistically consistent in the presence or absence of medications and in populations resistant to the use of three medications.
BACKGROUND: NHS services are under considerable strain, leading to delays in treatment that can cause increased harm to patients. Urgent and emergency care (UEC) centres are at the forefront of the NHS and as such are susceptible to these system pressures. Virtual wards (VWs) have been identified as a potential tool to help ease system pressures. A VW enables patients who would otherwise be in hospital to receive acute care and treatment in their usual place of residence or other community settings. VWs can facilitate an early discharge from a hospital setting (step-down care) or provide an alternative to hospital admission (step-up care). There is some evidence supporting the safety of VWs, but further evaluation is needed in the consideration of clinical efficacy and cost-effectiveness. This research evaluates why evidence generation in the field of VWs is challenging and discusses important factors for future evaluation, with a focus on the key pathways outlined by the NHS.
METHODS: A pragmatic literature review of multiple areas of VWs was undertaken to understand the fast-moving evidence within VWs. The pragmatic literature review was focused on safety, clinical effectiveness ,and cost-effectiveness outcomes. Gap analysis was then undertaken based on the evidence identified, to understand where future evidence generation should be prioritised. This included identifying the issues with generating evidence for VWs. The authors had previously led an early value assessment (EVA) for NICE as part of their role as an external assessment group, which involved patient and clinical engagement. The specific EVA was focussed on VW for acute respiratory infection. The authors used this experience to help inform the pragmatic searches and key areas for gap analysis.
RESULTS: The evidence base for VWs suggests that they are potentially safe and effective, although, the evidence is limited. The evidence supporting the clinical effectiveness of VWs varies between the patient cohort analysed and whether the ward provides step-up, step-down or mixed model care. Most evidence identified was non-comparative, or underpowered to capture some of the relevant outcomes. Case studies were reported for VWs implementation in the NHS in England. However, these studies lacked peer review, involved small sample sizes and lacked transparency surrounding the costing of VWs. Key issues were identified with the evaluation of VW, including the consideration of varying components which make up a tech-enabled VW, a lack of comparison to manual forms of a VW, accounting for different populations and subgroups associated with the use of VWs, and how a VW may distort the population when compared with standard care. Key areas for future evidence generation included prospective cohort studies to better understand clinical and resource use outcomes, identifying the impact of differences in VW features, as well as studies which can identify the true resource use associated with implementing and running a VW.
IMPLICATIONS & LIMITATIONS: Technology-enabled VWs are complex care systems. There is some evidence that they may provide resource benefits to the NHS. However, unless they are appropriately evaluated, we may not understand the true clinical efficacy, safety risks and costs associated with VW implementation. This is especially pertinent given the rapid expansion of VW facilities across the NHS. Future evaluation will be key to determine whether VWs are an effective alternative to hospital care, the features that may drive this effectiveness, and how these features could be harnessed to optimise effectiveness. Limitations of this research include the pragmatic nature of the review, as well as the generalisability of some of the identified evidence for VWs.
